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ACT’s Stem Cell Clinical Trial Expanding to Bascom Palmer

Woman looking through microscopeI am pleased to report that Advanced Cell Technology (ACT) just received institutional review board approval to add Bascom Palmer Eye Institute in Miami, Florida, as a clinical trial site for its stem-cell-derived treatment for people with dry age-related macular degeneration (AMD). Bascom Palmer has been ranked by U.S. News & World Report as the best ophthalmology hospital in the U.S. for the last eight years, so they are no doubt a valuable addition to the study.

ACT’s clinical trial of its treatment for dry AMD is also taking place at UCLA and Wills Eye Institute in Philadelphia.

The clinical trial of the same treatment for Stargardt disease is underway at UCLA and Wills, as well as Grampian University Hospitals and Moorfields Eye Hospital in the U.K.

Earlier this year, ACT announced encouraging results for the first two trial participants treated, one with dry AMD and the other with Stargardt disease. There were no adverse events, and the cells provided modest vision improvement.

The expansion of these studies to additional sites and the treatment of more participants will provide more information about the treatment’s potential for saving vision. I look forward to reporting additional clinical trial results as they are made available.

If you haven’t done so already, make sure you read Dr. David Gamm’s guest blog post on his outstanding stem cell research and the enormous potential it has as both a treatment and a research tool.

14 Responses to 'ACT’s Stem Cell Clinical Trial Expanding to Bascom Palmer'

  1. MARK MAURER says:

    Could this clinical trail also help my wife who is blind due to an atrophied retina in her right eye and optical nerve damage in her left eye. If clinical trails are being conducted could her name be added to the list asap? Please advise


  2. Maria Dorst says:

    Which criteria has to be met in order to be admitted to this trial. My father has dry AMD, however he does not live in the U.S.



    • adriana Tietzsch says:

      Do you have any treatments for kids?? age 10 with stargardt diseace? Canada,Alberta

      • EyeOnTheCure says:

        Hi Adriana, unfortunately, there are no FDA-approved treatments for patients with Stargardt disease, regardless of the age. However, it is encouraging to know that the Foundation Fighting Blindness is partnering with Sanofi Pharmaceuticals on a gene therapy clinical trial for Stargardt disease. If your child is 6 years of age or older, he or she can participate. For more information on this trial, see the following link: Before anyone can apply to participate in any gene therapy trial they must first obtain a molecular (genetic) diagnosis. For information on genetic testing, please see the following web link to download a PDF document:
        I highly recommend that you enroll your son or daughter in “My Retina Tracker”, a free registry that helps people find out about clinical trials that are recruiting. For more information on “My Retina Tracker” please see the following web link:
        You may also be interested to know that there is a “Stargardt – Macular Degeneration” Facebook page where you can communicate with other people affected by Stargardt disease. Here is the link:
        Finally, below is a list of pharmaceutical / biotech companies that are developing therapies for Stargardt Disease:
        Acucela,( is a Seattle-based biotechnology company that is developing several drugs for retinal diseases such as AMD, dry eye, diabetic retinopathy, retinopathy of prematurity and Stargardt disease. Acucela’s visual cycle modulators (VCM) reduce the activity of the rod visual system — in essence, “slowing it down” and reducing the metabolic load on the retina. Reducing the speed of the visual cycle has been shown to protect the retina from light damage and reduce the accumulation of retinal-related toxic by-products, including A2E, which is implicated in both Stargardt disease and dry AMD. The Company’s lead investigational compound (Emixustat™) is currently in Phase 3 trials for dry AMD. Once approved by the FDA, Emixustat could be prescribed for Stargardt disease.

        Ocata Therapeutics (, a Santa Monica-based biotechnology company, has developed an RPE cell line that is derived from embryonic stem cells (ESC). Studies have shown that the subretinal transplantation of ESC-RPE cells in a rat RP model resulted in 100% visual function rescue. Functional rescue was also achieved in the Stargardt mouse model with near-normal functional measurements recorded at more than 70 days. The RPE cell transplantation studies are now in Phase 2 human clinicals. Here is the link to the Clinical Trials.Gov recruitment web page:
        *Note: It is not known how long the transplanted RPE cells will last in a human patient with Stargardt disease. Unless gene or pharmaceutical-based augmentation treatment is coupled to the RPE transplant, toxic A2E will continue to be produced and eventually kill the RPE cells.

        Alkeus, ( Alkeus has developed a form of vitamin A that upon light interaction, does not form toxic vitamin A metabolites and A2E. Alkeus’ lead compound, ALK-001, is an oral compound with a well-understood mechanism of action. ALK-001 was specifically designed to treat Stargardt disease by preventing the formation of these toxic vitamin A dimers in the eye. Alkeus is currently recruiting patients for a Phase 2 human clinical trial. Here is the link to the clinical recruitment page:
        Visum Therapeutics, ( Visum Therapeutics is developing a small molecule therapy to treat Stargardt’s disease. Visum’s novel approach proposes to develop drugs that will temporarily control levels of A2E in the eye and preserve the natural vision cycle, leading to a therapeutic treatment. Visum has discovered a unique chemical approach to sequester rather than eliminate A2E. Through this process, 25 diverse FDA approved drugs demonstrating both mechanistic and in vivo efficacy have been identified. Visum has identified a lead compound, VSM 20R, which is an enantiomer of an FDA approved drug that demonstrates complete retinal protection in preclinical studies. Visum plans to conduct Phase I and Phase II clinical trials in the near future.

        I hope you found this information useful and thank you for your support.

  3. My mother has dry AMD and I was wanting to know how she could get on the list for the trial study at Bascom Palmer Eye Institute. I look forward to hearing from you. Thank you

    • EyeOnTheCure says:

      Hi Debbie, thanks for commenting

      Here is a link to the clinical trial listing as well as contact information at Bascom-Palmer:

    • Jean Marie Estadt says:

      I am a very active seventy-six year old diagnosed with dry Macular Degeneration in July of 2012. I would be interested in being part of any current dry
      Macular Degeneration Trials,

      • EyeOnTheCure says:

        Dry AMD is most common type of macular degeneration and accounts for more than 90% of all AMD. In the dry form, the retinal pigment epithelial cells (RPE) sicken and die in the macula. These RPE cells support the light sensitive photoreceptor cells that are so critical to vision. Dry AMD is often referred to as atrophic AMD and is characterized by the presence of drusen within the macula. To read more about AMD please see the following web link:
        The government website: http://WWW.CLINICALTRIALS.GOV lists all of the “legitimate” clinical trials taking place. If you query in “DRY AMD”, you will see approximately 30 trials that are recruiting patients for clinical trials. Here is the web link:
        You will need to look at each trial to see whether or not you fit the inclusion and exclusion criteria. Some trials pay for travel and some do not and each trial should list a phone number where you can call for more information.

  4. john graybeal says:

    Would love to receive your news letter regarding Macular degeneration dry

  5. Karen Wilkins says:

    My son has Leber’s disease which has affected his eyesight greatly. Are there any studies that he could participate in? It has been about 9 1/2 years since he lost his sight.

    • EyeOnTheCure says:

      You should consider genetic testing for your son to try and identify the mutant gene responsible for causing his disease. Currently, there are around a dozen different genes that cause LCA.
      If the gene is identified, medical databases such as PubMed ( can be searched to identify any research that is being conducted. With a molecular diagnosis, your son may also qualify for gene therapy trials that are taking place. For information on genetic testing, please see the following web link to download a PDF document:

      Whether the disease gene is identified or not, one should still consider participating in FFB’s “My Retina Tracker”, a free registry that monitors clinical trials that are recruiting for various retinal diseases. For more information on “My Retina Tracker” please see the following web link:

      It may also be helpful to periodically check the website: http://WWW.CLINICALTRIALS.GOV which is maintained by the National Institutes of Health and contains a searchable list of clinical trials for most known diseases. Each clinical trial listing will provide you with information on what the study is about, the requirements for participating and contact information.

  6. Bobbie Jaynes says:

    I am loosing vision rapidly due to retina separation and lattice degeration and myopia in right eye left eye is completely gone no vision i also have dry eyes and more complications un diagnoised at this time…waiting for bliwaiting for blimd services to get on the ball to send me for evaluations i would like to see if i can be a part of clinical case study trials if possible how and who do i contact i live in Spring Hill fl.

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